Status:
COMPLETED
Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
Lead Sponsor:
HemaQuest Pharmaceuticals Inc.
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
12-60 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
Eligibility Criteria
Inclusion
- Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
- Between 12 and 60 years of age, inclusive
- At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
- Screening (untransfused) HbF level \>/= 2% as analyzed by a central laboratory
- If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
- Able and willing to give informed consent
- If female, must have a negative serum pregnancy test within 7 days of dosing
- If female, must not be of childbearing potential defined as post-menopausal by at least 2 years or surgically sterile, or must agree to use a medically accepted form of contraception throughout the study
- If the sexual partner of a male subject is a WCBP, she must agree to use a medically accepted form of birth control for themselves or their partner throughout the study
- In the view of the Investigator, able to comply with necessary study procedures
Exclusion
- Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
- Participation in a regular blood transfusion program
- More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
- An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
- Pulmonary hypertension requiring oxygen
- QTc \> 450 msec on screening
- Alanine transaminase (ALT) \> 3X upper limit of normal (ULN)
- Creatinine phosphokinase (CPK) \> 20% above the ULN
- Serum creatinine \>1.2 mg/dL
- An acute illness (e.g., febrile, gastrointestinal \[GI\], respiratory) within 72 hours prior to receiving first dose of study medication
- History of syncope, clinically significant dysrhythmias or resuscitation from sudden death
- Chronic opiate use which, in the view of the Investigator, could confound evaluation of an investigational drug
- Current abuse of alcohol or drugs
- Received another investigational agent within 4 weeks, or 5 half-lives, whichever is longer, prior to administration of study medication
- Currently pregnant or breast feeding a child
- Known infection with HIV-1
- Infection with hepatitis B or hepatitis C such that patients are currently on therapy or will be placed on therapy during the trial
Key Trial Info
Start Date :
March 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT00842088
Start Date
March 1 2009
Last Update
August 1 2011
Active Locations (9)
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1
Trialogic Research
Madison, Alabama, United States, 35758
2
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
3
Century Clinical Research, Inc.
Daytona Beach, Florida, United States, 32117
4
Medical College of Georgia
Augusta, Georgia, United States, 30912